PipelineRSSMay 5
FDA grants fast track status to 1-time stem cell therapy for hemophilia A
The FDA has given special fast-track status to a new stem cell therapy being developed by Expression Therapeutics for hemophilia A, a bleeding disorder. This designation means the therapy can be reviewed faster than usual. The therapy is designed to be a one-time treatment that could provide lasting relief from hemophilia A symptoms.
WHY IT MATTERSIf successful, this one-time stem cell therapy could eliminate the need for regular blood clotting factor infusions that hemophilia A patients currently require multiple times per week.